Right-to-try legislation: Primum non nocere just went out the window
Folks sometimes ask why I have no respect for most politicians, and I’m always surprised.
I hold the members of the modern second estate — in other words, those who have generally replaced the nobility in modern population governance — to a high standard of accountability. This leads me to respect anyone who seeks election to make a positive impact on society, who shows courage and stands up for high principle and transparency whenever feasible, and who is a consistent advocate for the welfare of the whole population, including those who are less enfranchised. I suppose that is why I have little respect for most politicians!
The recent federal “right-to-try” legislation represents another sad episode in U.S. politics, which will likely cause more harm than good, and represents pandering to a small section of the electoral public and lobby groups. The politicians who sponsored this bill (H.R. 878), although probably well intentioned, simply did not do their homework properly. Once again, those without appropriate training or knowledge have created feel-good federal legislation without sufficient caveats to ensure benefit or safety, with little chance of helping patients and with a serious possibility of causing major harm.
In brief, I’m concerned about the following:
- The legislation allows treatment for any patient with a terminal illness, without medically defining what “terminal” actually means in this context, nor defining any aspect of mental, emotional, biochemical or physical fitness to endure the potential toxicities of the agent under consideration. The approach to phase 1/phase 2 — early phase — clinical trials has evolved over the past 60 years and is mostly predicated on common sense and experience-driven rules to maximize safety. A heavily pretreated patient who is dying, with a performance status of 4, with accompanying liver and bone marrow dysfunction and widespread metastases, has no realistic chance of gaining real benefit from any novel therapy available today. By contrast, that patient has an extraordinary chance of sustaining horrendous toxicity, a reduction in active life expectancy, and a dramatic increase in hospitalization if treated inappropriately with an experimental agent with incomplete definition of toxicity and efficacy.
- The legislation defines that treatment must be given to a patient who “has received a certification from a physician, who is in good standing with the physician’s certifying organization or board.” By this criterion, I — a trained and certified medical oncologist — should be entitled to perform a retroperitoneal lymph node dissection in a postchemotherapy testis cancer patient or a liver transplant in a 5-year-old patient, despite not having any specific qualifications to allow me to do either of these procedures with safety. It is ridiculous to believe that a physician with no experience in phase 1/phase 2 cancer trials should be entitled to order the delivery of an investigational drug for any patient — and certainly not one with multisystem failure, near death. The reality is that we live in an era of subspecialization, and this has led to improvement in outcomes because relevant specialists actually know the strengths, limitations and toxicities of the treatments that they use. This law simply ignores that and, thus, will create a type of medical anarchy while increasing patient risk.
- The reality of addressing cost, and who pays for medical care — despite much rhetoric — seems to be alien to Capitol Hill. Its inhabitants seem very happy to create scenarios that will dramatically escalate cost — eg, the panoply of new quality improvement and documentation rules that don’t connect to true health care improvement or the requirement to produce electronic physician order entry for medications when most available electronic medical record systems are so heavily flawed as to be unusable for chemotherapy prescription. Now they have added right-to-try legislation to the list but appear less keen to work out who should actually be responsible for paying for the treatments that will be used. In creating this new law, the sponsors have not deigned to work out who will carry the can — for example, if one is foolish enough to prescribe chimeric antigen receptor T-cell therapy for terminally ill patients, those lucky enough to survive the procedure (and the potential of cytokine release syndrome) could face medical, hospital and drug bills in excess of $1,000,000. More likely in this setting is the victim of a Hail Mary attempt with a checkpoint inhibitor, which could kill the patient, would be unlikely to be effective against the cancer and could produce a bill of more than $500,000! Do the sponsors of the bill see it as reasonable for the patient and family to carry this cost? How about the health insurance company, which never pays for experimental drugs? Maybe the pharmaceutical manufacturer — they don’t even have enough money available to ensure a continuous supply of cheap, generic drugs, so this seems the least likely of all. As of June — weeks after our political friends had voted in favor of this bill — no estimate of its costs had been published by the Congressional Budget Office! Talk about a blank check of taxpayer money to be expended with little potential for benefit and great potential for harm.
Let’s understand what this means. More than 600,000 people will die of cancer in 2018. Hopefully most of these unfortunate souls will be too sensible to leverage this legislation, and instead will consider current standards of investigational care or the use of comfort care and hospice.
However, if only 1% of these terminally ill patients (n = 6,000) take advantage of this new law, and each one convinces some idiot to try desperation treatment with a checkpoint inhibitor, based on current costs, this could produce a collective bill of more than $1,800,000,000! If the Goldwater Institute and other advocates push this concept, it could cost 10 to 50 times more — and that would still only encompass half of the patients who would ultimately die of cancer this year.
If it were likely they would truly achieve meaningful benefit from this approach, it might be worthy of consideration. However, the bottom line is that right-to-try legislation might prolong life significantly in fewer than 3% to 5% of cases, and it could shorten life and increase pain and suffering and expense in a much larger proportion.
Seems like a really bad deal to me.
For more information:
Derek Raghavan, MD, PhD, FACP, FRACP, FASCO, is HemOnc Today’s Chief Medical Editor for Oncology. He also is president of Levine Cancer Institute at Atrium Health. He can be reached at email@example.com.
Disclosure: Raghavan reports no relevant financial disclosures.